Doctors inject lab-grown genes coupled with viruses to good result
The equipment pictured here is likely more elaborate than that which the doctor used to treat me upon my return from Belarus. As I was preparing to travel to the Minsk airport, Wednesday morning August 1st, I had what turned out to be an irregularity of some kind with my appendix. Laid on the table Thursday evening after 14 hours of flight, laparoscopic equipment enabled the surgeon to go on a search and rescue mission which ended with a healthier Ray. What is pictured here is more complex because at the microscopic level of gene therapy, used to treat the blindness of a thirteen year old boy.
This gene therapy, called gene-replacement therapy, is designed to replace the mutated genes with genes that work, and accordingly, improve the patient’s degenerating eyesight. Synthetic genes are first grown in a lab and then inserted into viruses commonly found in the human eye. These are viruses which naturally live in our eyes and are harmless. The mission of the implanted viruses is to invade eye cells and infuse genetic material into them. To accomplish the beneficent mission, the viruses are carefully placed in the patient’s retina.
The gene replacement effort is designed to replace the RPE65 gene in the retina. This mutated gene fails over time to produce the needed proteins required for clear vision. The virus becomes a carrier for the synthetically created gene set to replace the faulty gene. The virus receives the synthetic gene and the ensuing material is placed into the retina. Because stronger than the failing gene, the inserted synthetic gene replaces the failing gene.
This is the first FDA approved gene replacement therapy for this incurable disease. No other meaningful treatments were available, so the FDA approved the replacement therapy more rapidly than under normal conditions. The rapid evaluation and approval of this remarkable gene replacement therapy was exercised under a program called Breakthrough Therapy and Priority Review.
With a cost of $425,000 per eye, there is a very limited target audience. However, to have developed the therapy conceptually opens the door for standardization and insurance approval at a lower level eventually. Currently, those having developed the procedure are working with insurers to enable acceptable payments
